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Actelion has announced that Zavesca (miglustat) has been approved in the European Union for the treatment of progressive neurological manifestations in adult patients and pediatric patients with Niemann-Pick type C disease (NPC).
Zavesca is the first treatment to be approved for patients with Niemann-Pick type C disease, a very rare, invariably progressive and eventually fatal neurodegenerative genetic disorder affecting both children and adults.
Zavesca (100 mg miglustat) is already indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease. Zavesca may only be used in the treatment of type 1 Gaucher patients for whom enzyme replacement therapy is unsuitable.
Jean-Paul Clozel, chief executive officer of Actelion commented: “I am very proud that Actelion – together with the scientific community – has been able to demonstrate the role of Zavesca in reducing the progression of clinically relevant neurological symptoms in patients with NPC. I would like to thank both the patients and their families who, over the years, have been involved in our clinical program with so much dedication, as well as all the clinical experts for their continuous support. Actelion will continue to support the rare disease community in its efforts to advance science and medicine for the patient”.
Ed Wraith, Royal Manchester Children’s Hospital, commented: “For the first time we have an approved therapy for NPC. The data on the effects of treatment with Zavesca obtained in a clinical trial and in a retrospective cohort study consistently showed a favorable clinical response. As a treating physician I am acutely aware of the importance of reducing progression of neurological symptoms.”
Regulatory proceedings to extend the use of miglustat in patients with NPC are ongoing in other territories worldwide.
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